Please use this identifier to cite or link to this item: https://dora.health.qld.gov.au/qldresearchjspui/handle/1/4666
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dc.contributor.authorGauld, L.en
dc.contributor.authorChacko, A.en
dc.contributor.authorSly, P. D.en
dc.contributor.authorDeegan, S.en
dc.date.accessioned2022-11-07T23:55:07Z-
dc.date.available2022-11-07T23:55:07Z-
dc.date.issued2020en
dc.identifier.citation201, (1), 2020en
dc.identifier.otherRISen
dc.identifier.urihttp://dora.health.qld.gov.au/qldresearchjspui/handle/1/4666-
dc.description.abstractRationale: Nusinersen is widely used for Spinal Muscular Atrophy (SMA), however respiratory effects are largely unknown. Aim: Assess change in respiratory function in pediatric SMA during first year of nusinersen treatment and compare to previous function. Methods: 12-month prospective study of all childhood SMA type 1- 3 in Queensland, Australia from June 2018. Muscle strength testing (Revised Hammersmith Functional Motor Scale for SMA, Hammersmith Infant Neurological Examination or Children's Hospital of Philadelphia Infant Test of Neurological Disorders) performed prior to commencement and 4 occasions during study period. Respiratory investigations also attained: Spirometry, Forced Oscillation Technique (FOT), Sniff Nasal Inspiratory Pressure (SNIP), Mean Inspiratory Pressure (MIPS), Mean Expiratory Pressure (MEPS), Lung Clearance Index (LCI). Full Polysomnography (PSG) conducted initially and study completion. Lung function 2 years prior to study commencement collected retrospectively. Lung function decline and PSG parameters pre- and post-treatment compared. Muscle strength responders/non-responders compared for respiratory function change. Results: 31 children (18 males, 0.25-18.8 years) included; 6 (19%) SMA type 1, 16 (52%) type 2 and 9 (29%) type 3. For all lung function parameters, stability noted throughout first year treatment (Table 1). Type 2 annual rate of decline in Forced Vital Capacity (FVC) z-score reduced from -0.5 per annum (p=0.001, 95%CI 0.26-0.72) to +0.002 (p=0.5, 95%CI -0.08-0.006), p=0.009; for type 3 SMA,-0.4 per annum (p=0.008 95%CI 0.1-0.6) to -0.001 (p=0.4, 95%CI -0.005-0.002), p=0.3. Mean total apnea-hypopnea index (AHI) reduced following treatment for type 1 [12.3 events/hour to 3.7], type 2 [4.5 (SD 3.0 95% CI 1.8-7.3) to 2.9 (SD 3.0 CI 0.1-5.6) p=0.1] and type 3 [5.3 (SD 2.2 95% CI0.14-10.8) to 2.5 (SD 19.98 95%CI 4.34) p=0.4]. Motor muscle response attained in 5/11 (45%) type 2, 6/9 (67%) type 3. No change in FVC z-score between responders/non-responders (FVC z-score 0.5 p=0.5 95%CI -1.2-2.1 r<0.001/0.1 p=0.9 95%CI -2.4-2.6 r=0.01 respectively). Conclusion: Nusinersen halts respiratory function decline seen before treatment and stabilizes lung function during first treatment year in type 2 and 3 SMA, and seen even in those without peripheral muscle strength response. Improvements in sleep disordered breathing in all types SMA seen in first year of treatment.L6323763332020-07-27 <br />en
dc.language.isoenen
dc.relation.ispartofAmerican Journal of Respiratory and Critical Care Medicineen
dc.titleStabilization of respiratory function in pediatric spinal muscular atrophy treated with nusinersenen
dc.typeArticleen
dc.subject.keywordsclinical articleen
dc.subject.keywordsconference abstracten
dc.subject.keywordscontrolled studyen
dc.subject.keywordsdrug therapyen
dc.subject.keywordsforced oscillation techniqueen
dc.subject.keywordsforced vital capacityen
dc.subject.keywordshumanen
dc.subject.keywordsinfanten
dc.subject.keywordsKugelberg Welander diseaseen
dc.subject.keywordslung clearanceen
dc.subject.keywordsmaleen
dc.subject.keywordsmuscle strengthen
dc.subject.keywordsneurologic examinationen
dc.subject.keywordsapnea hypopnea indexen
dc.subject.keywordspolysomnographyen
dc.subject.keywordsprospective studyen
dc.subject.keywordsQueenslanden
dc.subject.keywordsretrospective studyen
dc.subject.keywordssleep disordered breathingen
dc.subject.keywordsspirometryen
dc.subject.keywordsWerdnig Hoffmann diseaseen
dc.subject.keywordsyoung adulten
dc.subject.keywordsadulten
dc.subject.keywordsnusinersenacute lymphoblastic leukemiaen
dc.subject.keywordsPennsylvaniaen
dc.subject.keywordschilden
dc.relation.urlhttps://www.embase.com/search/results?subaction=viewrecord&id=L632376333&from=exporten
dc.identifier.risid726en
item.openairecristypehttp://purl.org/coar/resource_type/c_18cf-
item.cerifentitytypePublications-
item.fulltextNo Fulltext-
item.grantfulltextnone-
item.openairetypeArticle-
item.languageiso639-1en-
Appears in Sites:Children's Health Queensland Publications
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